News from the FDA/CDC

FDA approves anemia treatment for transfusion-dependent beta thalassemia patients


The Food and Drug Administration has approved the first treatment for anemia in adults with transfusion-dependent beta thalassemia.

A stamp saying "FDA approved." Olivier Le Moal/Getty Images

Luspatercept-aamt (Reblozyl) is an erythroid maturation agent that reduced the transfusion burden for patients with beta thalassemia in the BELIEVE trial of 336 patients. In total, 21% of patients who received luspatercept-aamt achieved at least a 33% reduction in red blood cell transfusions, compared with 4.5% of patients who received placebo, according to the FDA.

Common side effects associated with luspatercept-aamt were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness. Patients taking the agent should be monitored for thrombosis, the FDA advised.

Celgene, which makes luspatercept-aamt, said the agent would be available about 1 week following the FDA approval.

The FDA is also evaluating luspatercept-aamt as an anemia treatment in adults with very-low– to intermediate-risk myelodysplastic syndromes who have ring sideroblasts and require red blood cell transfusions. The agency is expected to take action on that application in April 2020.

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